MigALastat Therapy Adherence Among FABRY Patients: A Prospective Multicentral Observational Study (MALTA-FABRY)

Wuerzburg University Hospital

Status: Enrolling

Conditions: Fabry Disease; Adherence, Medication; Quality of Life; Rare Diseases

Study type: Observational

Funder types: Other

Identifiers

NCT03683966

AT-IIP-2018

Details and patient eligibility

About

This study evaluates adherence to the oral chaperone therapy migalastat in patients with Fabry disease.

Full description

Fabry disease is a rare disease and part of the group of lysosomal storage disorders. Since 2016, chaperone therapy as a new therapeutic approach is available.

This study is a prospective cohort study and observes patients under therapy with migalastat. This study is suggested to help estimating the adherence of the oral therapy.

All patients in treatment with migalastat in the Fabry Center Wuerzburg (FAZiT) and selected patients of other cooperating Fabry Centers are included in this study if informed consent is provided.

Enrollment: 30 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Fabry disease (genetically confirmed)
• Signed informed consent
• 18 years and older

Exclusion criteria

• No informed consent
• Withdrawal of informed consent